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A multicenter open-label extension study of intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A.
Wijburg, Frits A; Whitley, Chester B; Muenzer, Joseph; Gasperini, Serena; Del Toro, Mireia; Muschol, Nicole; Cleary, Maureen; Sevin, Caroline; Shapiro, Elsa; Alexanderian, David.
Affiliation
  • Wijburg FA; Academic Medical Center, Amsterdam, the Netherlands. Electronic address: f.a.wijburg@amc.uva.nl.
  • Whitley CB; University of Minnesota, Minneapolis, MN, USA. Electronic address: whitley@umn.edu.
  • Muenzer J; University of North Carolina, Chapel Hill, NC, USA. Electronic address: muenzer@med.unc.edu.
  • Gasperini S; Fondazione MBBM, San Gerardo Hospital, Monza, Italy. Electronic address: s.gasperini@hsgerardo.org.
  • Del Toro M; Hospital Vall D'Hebron, Barcelona, Spain. Electronic address: mdeltoro@vhebron.net.
  • Muschol N; University Medical Center Hamburg-Eppendorf, Department of Pediatrics, Hamburg, Germany. Electronic address: muschol@uke.de.
  • Cleary M; Great Ormond Street Hospital, London, UK. Electronic address: Maureen.Cleary@gosh.nhs.uk.
  • Sevin C; Unit, GHU Paris-Sud - Hôpital de Bicêtre, Le Kremlin Bicêtre, Paris, France. Electronic address: caroline.sevin@inserm.fr.
  • Shapiro E; University of Minnesota, Minneapolis, MN, USA. Electronic address: shapi004@umn.edu.
  • Alexanderian D; Takeda Pharmaceutical Company Limited, Lexington, MA, USA. Electronic address: david.alexanderian@takeda.com.
Mol Genet Metab ; 134(1-2): 175-181, 2021.
Article in En | MEDLINE | ID: mdl-34247932
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Sulfatases / Central Nervous System / Mucopolysaccharidosis III Type of study: Clinical_trials Limits: Child, preschool / Female / Humans / Infant / Male Language: En Journal: Mol Genet Metab Journal subject: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Year: 2021 Document type: Article
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Sulfatases / Central Nervous System / Mucopolysaccharidosis III Type of study: Clinical_trials Limits: Child, preschool / Female / Humans / Infant / Male Language: En Journal: Mol Genet Metab Journal subject: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Year: 2021 Document type: Article